lunsotogene parvec

Lunsotogene parvec (brand name: Otarmeni) is an adeno-associated virus (AAV) vector-based gene therapy developed by Regeneron Pharmaceuticals for the treatment of pediatric and adult patients with severe-to-profound sensorineural hearing loss caused by biallelic variants in the OTOF gene. These variants result in the production of non-functioning otoferlin protein, which is essential for the transmission of auditory signals from hair cells to the auditory nerve. The therapy delivers a functional copy of the OTOF gene directly to cochlear hair cells via a single intracochlear infusion. Expression is restricted to hair cells using a proprietary cell-specific Myo15 promoter. It received FDA accelerated approval in April 2026, becoming the first gene therapy approved for genetic hearing loss.

Brand names

Otarmeni

Other names

lunsotogene parvec-cwha